Clinical Trials in Thalassemia Cell and Gene Therapy Grants

The Cooley’s Anemia Foundation invites national and international applicants to apply for grants to facilitate clinical trials in Cell and Gene Therapy to advance a cure for thalassemia.   The purpose of this initiative is to speed the application of recent translational advances in cell and gene therapy to clinical trials. Both phase I (safety) and phase II (efficacy) trials are eligible for support.

Funding and candidate eligibility

Applicants should state explicitly the status of their proposed trials; for example, whether the trial has been approved by the Recombinant DNA Advisory Committee (RAC), or Food and Drug Administration (or equivalent processes at non-US sites), or whether review meetings at regulatory agencies have been scheduled. 

Studies proposed for this RFA must be accompanied by a realistic timeline of progress to human trials. The Foundation recognizes that this timeline can be long.   While the funds may be used toward any aspect of launching a human gene therapy trial, the Foundation will request careful documentation of other support of the investigator(s) and the project, and a thorough justification of the allocation of costs to various support mechanisms. This will best enable reviewers to assess feasibility of the proposed project. Examples of expenses allowed will include:

• Clinical-grade vector production and quality control • Research subject assessments • Late-preclinical studies and ex vivo studies on research subjects required for safety and efficacy assessments (including, but not limited to, evaluation of vector genome integration events, studies of transduction efficiency, studies of globin production by transduced hematopoietic stem cells from subjects) • Patient care costs directly related to the study • Research assistant or research nursing support.

Assurance must be given that adequate clinical and other facilities (including, where applicable, laboratory and vector production facilities) exist and are available to conduct the research project and, in case of investigations involving human subjects, a full human subject protection plan should be included as an appendix.

Total funding of up to $60,000 per year will be available (including indirect costs of 8%). With satisfactory evidence of progress and availability of funding grants may be renewed for a second year.  Excluded from funding in this grant are lodging and other personal costs for research subjects to come to a treatment center.

Support for investigator effort may not exceed 10% of the current NIH salary cap.  Animal studies, if any, must be directly relevant to the specific proposed clinical trial.

The research may not be conducted at a for-profit laboratory.

Application due date

The completed application is due February 4, 2013 and a PDF format of the application should be emailed to:  

Application  forms and instructions for this grant can be downloaded by clicking here.

Grant award will be announced in June, 2013.


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